Abstract：
:The clustered regularly interspaced short palindromic repeat (CRISPR) has great potential to revolutionize biomedical research and disease therapy. The specific and efficient genome editing strongly depends on high efficiency of delivery of the CRISPR payloads. However, optimization of CRISPR delivery vehicles still remains a major obstacle. Recently, various non-viral vectors have been utilized to deliver CRISPR tools. Many of these vectors have multi-layer structures assembled. In this review, we will introduce the development of CRISPR-Cas9 systems and their general therapeutic applications by summarizing current CRISPR-Cas9 based clinical trials. We will highlight the multi-layer nanoparticles (NPs) that have been developed to deliver CRISPR cargos in vitro and in vivo for various purposes, as well the potential building blocks of multi-layer NPs. We will also discuss the challenges in making the CRISPR tools into viable pharmaceutical products and provide potential solutions on efficiency and biosafety issues.
journal_name
Adv Drug Deliv Revjournal_title
Advanced drug delivery reviewsauthors
Tang H,Zhao X,Jiang Xdoi
10.1016/j.addr.2020.03.001subject
Has Abstractpub_date
2021-01-01 00:00:00pages
55-78eissn
0169-409Xissn
1872-8294pii
S0169-409X(20)30013-2journal_volume
168pub_type
杂志文章,评审abstract：:Epilepsy is the most common serious chronic neurological disorder. Current data show that one-third of patients do not respond to anti-epileptic drugs (AEDs). Most non-responsive epilepsy patients are resistant to several, often all, AEDs, even though the drugs differ from each other in pharmacokinetics, mechanisms of...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2011.12.003
更新日期：2012-07-01 00:00:00
abstract：:This review presents a summary of the various types of cellular therapy used to treat spinal cord injury. The inhibitory environment and loss of axonal connections after spinal cord injury pose many obstacles to regenerating the lost tissue. Cellular therapy provides a means of restoring the cells lost to the injury a...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2007.08.028
更新日期：2008-01-14 00:00:00
abstract：:Tip-enhanced Raman scattering (TERS) has become a powerful tool for nanoscale structural analysis for several branches of organic, inorganic, and biological chemistry. This highly sensitive technique enables molecular characterization with a lateral resolution far beyond Abbe's diffraction limit and correlates structu...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2015.06.007
更新日期：2015-07-15 00:00:00
abstract：:The description of irregular particle morphology and powder properties may offer unique insights into the performance of materials intended for use in dry powder inhalers. A number of mathematically similar approaches have been used to study these phenomena, all based on evaluating data in terms of irregular oscillati...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(97)00508-5
更新日期：1997-06-09 00:00:00
abstract：:Vaccines are one of the most powerful technologies supporting public health. The adaptive immune response induced by immunization arises following appropriate activation and differentiation of T and B cells in lymph nodes. Among many parameters impacting the resulting immune response, the presence of antigen and infla...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.06.019
更新日期：2020-01-01 00:00:00
abstract：:Intravenous administration of low (milligram) doses of nanoparticulate materials in pigs can lead to acute cardiopulmonary, hemodynamic, hematological, biochemical and dermatological changes within minutes, mimicking the human infusion (or anaphylactoid) reactions to many state-of-the-art (nano)medicines and biologica...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.07.005
更新日期：2012-12-01 00:00:00
abstract：:An aging population in the developing world has led to an increase in musculoskeletal diseases such as osteoporosis and bone metastases. Left untreated many bone diseases cause debilitating pain and in the case of cancer, death. Many potential drugs are effective in treating diseases but result in side effects prevent...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.01.012
更新日期：2012-09-01 00:00:00
abstract：:Small G proteins or GTPases comprise a growing family of signal transduction molecules with inducible properties dependent upon reversible interactions with guanine nucleotides. Activation status of the proteins is characterized by preferential affinity for triphosphorylated guanine nucleotides, initiating signaling e...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(00)00050-8
更新日期：2000-06-30 00:00:00
abstract：:Cell-based therapy is emerging as a promising strategy for treating a wide range of human diseases, such as diabetes, blood disorders, acute liver failure, spinal cord injury, and several types of cancer. Pancreatic islets, blood cells, hepatocytes, and stem cells are among the many cell types currently used for this ...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2018.04.018
更新日期：2019-01-15 00:00:00
abstract：:The encouraging pace of discovery and development in the field of regenerative medicine holds tremendous potential for bringing therapies to the clinic that may offer meaningful benefit to patients, particularly in diseases with no or suboptimal therapeutic options. Academic researchers will continue to play a critica...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2016.10.001
更新日期：2017-12-01 00:00:00
abstract：:Cellular processes depend on the fidelity of intracellular membrane traffic. Lipids, proteins, receptor ligands and solute molecules are trafficked to distinct compartments within the cell through both the biosynthetic and endocytic pathways. An appreciation of these pathways is vital for a complete understanding of i...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2004.05.003
更新日期：2005-01-02 00:00:00
abstract：:Lipid-coated microbubbles represent a new class of agents with both diagnostic and therapeutic applications. Microbubbles have low density. Stabilization of microbubbles by lipid coatings creates low-density particles with unusual properties for diagnostic imaging and drug delivery. Perfluorocarbon (PFC) gases entrapp...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2003.12.006
更新日期：2004-05-07 00:00:00
abstract：:Thioredoxin 1 (Trx 1) is a redox-active small protein ubiquitously present in human body. It is one of the defensive proteins induced in response to various stress conditions. In addition to its anti-oxidative effect by dithiol-disulfide exchange in its active site, Trx 1 has anti-apoptotic and anti-inflammatory effec...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2009.01.003
更新日期：2009-04-28 00:00:00
abstract：:The ever increasing improvements of pharmaceutical formulations have been often obtained by means of the use of hydrogels. In particular, environmentally sensitive hydrogels have been investigated as "smart" delivery systems capable to release, at the appropriate time and site of action, entrapped drugs in response to...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2013.04.002
更新日期：2013-08-01 00:00:00
abstract：:Modern pharmaceutical delivery systems are intended to produce plasma drug concentration versus time profiles that result in optimum therapeutic efficacy and a minimum of drug concentration-dependent adverse effects. To accomplish this requires that the drug delivery rate and temporal profile be based on the pharmacok...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(98)00028-3
更新日期：1998-09-07 00:00:00
abstract：:Skin pigmentation is a result of melanin produced by melanocytes in the epidermis. Melanocyte activity, along with the type and distribution of melanins, is the main driver for diversity of skin pigmentation. Dark melanin acts to protect against the deleterious effects of ultraviolet (UV) radiation, including photo-ag...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.02.002
更新日期：2020-01-01 00:00:00
abstract：:Synthetic micro/nanomotors are tiny devices than can be self-propelled or externally powered in the liquid phase by different types of energy source including but not limited to: catalytic, magnetic or acoustic. Showing a myriad of mechanical movements, building block materials, sizes, shapes and propulsion mechanisms...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2015.09.004
更新日期：2015-12-01 00:00:00
abstract：:In recent years, nanotechnology has offered attractive opportunities to overcome the (bio)pharmaceutical drawbacks of most drugs such as low aqueous solubility and bioavailability. Among the numerous methodologies that have been applied to improve drug performance, a special emphasis has been made on those that increa...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2018.07.012
更新日期：2018-06-01 00:00:00
abstract：:The bloodstream is the main transporting pathway for drug delivery systems (DDS) from the site of administration to the intended site of action. In many cases, components of the vascular system represent therapeutic targets. Endothelial cells, which line the luminal surface of the vasculature, play a tripartite role o...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.06.013
更新日期：2020-01-01 00:00:00
abstract：:Infectious diseases are prevalent in the developing world and are one of the developing world's major sources of morbidity and mortality. While infectious diseases can initiate in a localized region, they can spread rapidly at any moment due to the ease of traveling from one part of the world to the next. This could l...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2009.11.015
更新日期：2010-03-18 00:00:00
abstract：:The objective of this review is to summarize the critical steps in intracellular trafficking and the principal factors involved in transgene expression by a comparison of viral and non-viral gene vectors. Intracellular trafficking of viral and non-viral gene vectors are reviewed kinetically as well as from the mechani...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/s0169-409x(01)00216-2
更新日期：2001-11-19 00:00:00
abstract：:Mesenchymal stem cells (MSC) are a promising tool for cell therapy, either through direct contribution to the repair of bone, tendon and cartilage or as an adjunct therapy through protein production and immune mediation. They are an attractive vehicle for cellular therapies due to a variety of cell intrinsic and envir...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2010.09.013
更新日期：2010-09-30 00:00:00
abstract：:Several synthetic and natural materials are used in soft tissue engineering and regenerative medicine with varying degrees of success. Among them, silkworm silk protein fibroin, a naturally occurring protein-based biomaterial, exhibits many promising characteristics such as biocompatibility, controllable biodegradabil...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2019.09.003
更新日期：2020-01-01 00:00:00
abstract：:Many kinds of inorganic nanoparticles (NPs) including semiconductor, metal, metal oxide, and lanthanide-doped NPs have been developed for imaging and therapy applications. Their unique optical, magnetic, and electronic properties can be tailored by controlling the composition, size, shape, and structure. Interaction o...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2012.08.015
更新日期：2013-05-01 00:00:00
abstract：:Despite the wide use of antibiotics, bacterial infection is still one of the leading causes of hospitalization and mortality. The clinical failure of antibiotic therapy is linked with low bioavailability, poor penetration to bacterial infection sites, and the side effects of antibiotics, as well as the antibiotic resi...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2014.02.002
更新日期：2014-11-30 00:00:00
abstract：:To guard against disease, mucosal surfaces of the intestinal, respiratory and urogenital tracts are protected by a carefully regulated system of defenses known as the mucosal immune system. The hallmark of mucosal immunity is secretory IgA which can prevent infection and remove antigen crossing the mucosal barrier. Ig...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(98)00042-8
更新日期：1998-12-01 00:00:00
abstract：:The incorporation of polymer-lipid conjugates, initially using PEG and subsequently other selected flexible, hydrophilic polymers, into lipid bilayers gives rise to sterically stabilized liposomes that exhibit reduced blood clearance and concomitant changes in tissue distribution largely because of reduced, but not el...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章
doi：10.1016/s0169-409x(97)00136-1
更新日期：1998-06-08 00:00:00
abstract：:Nanoparticles can provide effective control of the release rate and tissue distribution of their drug payload, leading to major pharmacokinetic and pharmacodynamic changes vis-à-vis the conventional administration of free drugs. In the last two decades, we have witnessed major progress in the synthesis and characteriz...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2020.05.012
更新日期：2020-01-01 00:00:00
abstract：:A mechanistic hypothesis is presented for how water-soluble guanidinium-rich transporters attached to small cargoes (MW ca. <3000) can migrate across the non-polar lipid membrane of a cell and enter the cytosol. Positively charged and water-soluble, arginine oligomers can associate with negatively charged, bidentate h...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2004.10.003
更新日期：2005-02-28 00:00:00
abstract：:Oral lipid-based drug delivery systems may include a broad range of oils, surfactants, and cosolvents. This diversity makes comparison of lipid-based formulations difficult. Although the relationship between formulation and drug absorption is understood at a conceptual level, performance in vivo cannot be predicted wi...
journal_title：Advanced drug delivery reviews
pub_type： 杂志文章,评审
doi：10.1016/j.addr.2007.10.010
更新日期：2008-03-17 00:00:00
